Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule.

Data from DELIVER and ACHIEVE Clinicals Trial to be Presented -- Presentation on ACHIEVE Trial in DM1 and Company Symposium to Feature Data on ...

CIS, Checklist Individual Strength FSHD, facioscapulohumeral muscular dystrophy HMSN-I, hereditary motor and sensory neuropathy type I MD, adult onset myotonic dystrophy SF-36, 36 item short form ...

candidate for myotonic dystrophy type 1 (DM1), and del-brax for facioscapulohumeral muscular dystrophy (FSHD). All three candidates are based on the same TfR1-targeting antibody component.

Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene, leading to muscle degeneration. Current treatments focus on maintaining muscle function, with emerging gene ...

The Janus Henderson Global Life Sciences Fund returned -11.16% and the MSCI World Health Care IndexSM returned -11.40%. Click ...

From left, Craig Meyer, Silvia Blemker and Joseph Hart are the University of Virginia’s Innovators of the Year. The UVA ...

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the global licensing and collaboration agreement with Sarepta Therapeutics (NASDAQ: SRPT) announced on November 26, 2024, has now ...